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Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

2023

Labrosse R et Al.

Blood 142, 1281-1296.

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Duchenne muscular dystrophy Infant Spinal muscular atrophy SMA-PME and Farber’s disease Myotubular myopathy Limb girdle muscular dystrophies Wiskott-Aldrich syndrome Fanconi’s anemia Chronic septic granulomatosis Cori Forbes disease Crigler-Najjar syndrome Pompe disease Leber Hereditary Optic Neuropathy

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